Aminothiadiazole, also known as NSC-4728; A-TDA; ADTA, is an inosine monophosphate dehydrogenase inhibitor potentially for the treatment of solid tumours. Aminothiadiazole is a synthetic derivative of nicotinamide adenine dinucleotide (NAD). Aminothiadiazole competitively inhibits inosine 5-monophosphate dehydrogenase, thereby disrupting the regulation of cell proliferation and differentiation in a number of cells. This agent is also a selective human adenosine A3 receptor antagonist.

Nitecapone, also known as OR-462, is a catechol-O-methyltransferase inhibitor potentially for the treatment of peptic ulcer. Nitecapone reduces development and symptoms of neuropathic pain after spinal nerve ligation in rats. Nitecapone reduces cardiac neutrophil accumulation in clinical open heart surgery. Nitecapone inhibits myeloperoxidase in vitro and enhances functional performance after 8 h of ischemia in experimental heart transplantation.

Methylthioninium Chloride, also known as TRx-014 and Methylene blue, has many uses in biology and chemistry; for example, it can be used as a stain and as a medication. Methylene blue should not be confused with methyl blue, another histology stain, new methylene blue, nor with the methyl violets often used as pH indicators. Methylthioninium Chloride is also tau protein inhibitor potentially for the treatment of Alzheimer's disease (AD) and Parkinson's disease.

Arasertaconazole is a sterol-14-alpha demethylation inhibitor potentially for treatment of vulvovaginal candcanidiasis.

Bufuralol, also known as Ro-34787, is a β-adrenoceptor antagonist with partial agonist activity and is potentially useful for the treatment of hypertension. It is metabolized by CYP2D6. Most beta blockers are aryloxypropanolamine-based. In this rare exception, the benzofuran oxygen is part of a ring instead of derived from the epichlorohydrin precursor.

Bicifadine HCl, also known as DOV-220075, CL 220,075, is a SNDR inhibitor potentially for the treatment of lower back pain. Bicifadine has a non-opioid, non-NSAID mechanism for the treatment of pain, which should have less abuse potential than opioid drugs and less propensity to cause gastric ulcers than NSAID drugs. While the drug is purported to be a serotonin (SERT) and noradrenaline transporter (NET) inhibitor, it also has effects at the dopamine transporter (DAT), effectively making it a broad-spectrum monoamine transporter inhibitor or "triple reuptake inhibitor."

Tesaglitazar, also known as AR-H-039242XX; AZ-242; ARH-039242XX, is a PPAR agonist potentially for the treatment of type 2 diabetes. The drug had completed several phase III clinical trials, however in May, 2006 AstraZeneca announced that it had discontinued further development.

Pregnanolone, also known as 3a5b-Pregnanolone; Kabi-2213, SKF 6455, Eltanolone, is a cell membrane modulator potentially for the treatment of pain. Pregnanolone is an endogenous neurosteroid that is biosynthesized from progesterone. It is a positive allosteric modulator of the GABAA receptor, as well as a negative allosteric modulator of the glycine receptor, and is known to have sedative, anxiolytic, anesthetic, and anticonvulsant effects. It was investigated for clinical use as a general anesthetic, but produced unwanted side effects such as convulsions on occasion, and for that reason was never marketed.

Sorbinil, also known as CP-45634, is an aldose reductase inhibitor potentially for the treatment of diabetes. Aldose reductase is an enzyme present in lens and brain that gets rid of excess glucose by converting it to sorbitol. Sorbitol accumulation can lead to the development of cataracts in the lens and neuropathy in peripheral nerves. Sorbinil has been shown to inhibit aldose reductase in human brain and placenta and calf and rat lens. Sorbinil reduced sorbitol accummulation in rat lens and sciatic nerve of diabetic rats orally administered 0.25mg/kg sorbinil.

Hydroxypropyl-beta-cyclodextrin, also known as Hydroxypropyl-beta-cyclodextrin; THPB; HP-beta-CD; HPbCD; HP-beta-CyD; VTS-270; HPbetaCD; HPbetaCyD, is a drug candidate potentially for the treatment of Niemann-Pick type C diseases. In April 2009, hydroxypropyl-beta-cyclodextrin (HPbCD) was first approved.

Acetylglucosamine or N-acetylglucosamine is potentially for the treatment of irritable bowel syndrome. N-Acetylglucosamine (GlcNAc) is a monosaccharide that usually polymerizes linearly through (1,4)-β-linkages. GlcNAc is the monomeric unit of the polymer chitin, the second most abundant carbohydrate after cellulose. In addition to serving as a component of this homogeneous polysaccharide, GlcNAc is also a basic component of hyaluronic acid and keratin sulfate on the cell surface. GlcNAc has been reported to be an inhibitor of elastase release from human polymorphonuclear leukocytes (range 8–17% inhibition), however this is much weaker than the inhibition seen with N-acetylgalactosamine (range 92–100%).

Licarbazepine, also known as BIA-2-005; GP-47779; LIC-477; LIC-477D; TRI-477, is a voltage-gated sodium channel blocker with anticonvulsant and mood-stabilizing effects that is related to oxcarbazepine. It is an active metabolite of oxcarbazepine. In addition, an isomer of licarbazepine, eslicarbazepine ((S)-(+)-licarbazepine), is an active metabolite of eslicarbazepine acetate. Oxcarbazepine and eslicarbazepine acetate are inactive on their own, and behave instead as prodrugs to licarbazepine and eslicarbazepine, respectively, to produce their therapeutic effects.

Pimagedine, also known as aminoguanidine, MDL-201228; YM-585; GER-11, is an AGE Inhibitor potentially for the treatment of diabetic nephropathy and diabetic retinopathy. Pimagedine is also a diamine oxidase and nitric oxide synthase inhibitor. It acts to reduce levels of advanced glycation end products (AGEs) through interacting with 3-deoxyglucosone.

Ganaxolone, also known as CCD 1042 and C1042, is a CNS-selective GABAA modulator that acts on well-characterized targets in the brain known to have anxiolytic and anticonvulsant effects. Ganaxolone protects against seizures in diverse animal models, including the pentylenetetrazol, 6 Hz and amygdala kindling models. Ganaxolone is a positive allosteric modulator of the action of the GABAA receptor and, unlike benzodiazepines, there does not appear to be tolerance to the anticonvulsant effects of ganaxolone.

Midafotel, also known as SDZ-EAA 494 and D-CPP-ene, is a potent, competitive antagonist at the NMDA receptor. It was originally designed as a potential therapy for excitotoxicity, epilepsy or neuropathic pain. It looked very promising in in vitro trials proving to be a potent competitive antagonist at the NMDA without affecting other receptors.

Mofegiline, also known as MDL-72974, or MDL-72974A (HCl salt), is a selective, irreversible inhibitor of monoamine oxidase B (MAO-B) and semicarbazide-sensitive amine oxidase (SSAO) which was under investigation for the treatment of Parkinson's disease and Alzheimer's disease, but was never marketed.

Ranirestat, also known as AS-3201; SX-3201, is an aldose reductase inhibitor being developed for the treatment of diabetic neuropathy by Dainippon Sumitomo Pharma and PharmaKyorin. It has been granted orphan drug status. The drug is to be used orally. Aldose reductase is an enzyme that catalyzes one of the steps in sorbitol (polyol) pathway which is responsible for formation of fructose from glucose. Aldose reductase activity is increased, parallel to glucose blood levels, in tissues that are not insulin sensitive, including lenses, peripheral nerves and renal glomeruli. Sorbitol does not diffuse through cell membranes easily and therefore accumulates in these tissues, causing osmotic damage, leading to retinopathy and neuropathy.

Pyridoxamine, also known as K-163, is one form of vitamin B6. Chemically it is based on a pyridine ring structure, with hydroxyl, methyl, aminomethyl, and hydroxymethyl substituents. It differs from pyridoxine by the substituent at the 4-position. The phenol at position 3 and aminomethyl group at position 4 of its ring endow pyridoxamine with a variety of chemical properties, including the scavenging of free radical species and carbonyl species formed in sugar and lipid degradation and chelation of metal ions that catalyze Amadori reactions.

Foropafant, also known as SR-27417; SR-27417A, is a potent platelet activating factor (PAF) inhibitor potentially for the treatment of asthma and allergic rhinitis (AR). SR 27417 inhibits in a dose-dependent manner the hypotensive effect of PAF in rats. It protected rats with an ED50 value of 6 +/- 2 micrograms/kg (n = 6), when given i.v., 1 min before PAF administration. PAF plays a major role in anaphylactic shock. SR 27417 may be an effective prophylactic as well as a potent curative drug in this pathology.

Remacemide, also known as PR-934423 and FPL 12924AA, is a low-affinity NMDA antagonist with sodium channel blocking properties. It has been studied for the treatment of acute ischemic stroke, epilepsy, Huntington's disease, and Parkinson's disease. Remacemide binds weakly and noncompetitively to the ionic channel site of the NMDA receptor complex. Remacemide binds both allosterically and in the channel. However, because remacemide binds so weakly to NMDAR, much of remacemide's in vivo effect against excitotoxicity is thought to be caused by its metabolic transformation to the more potent desglycine derivative FPL 12495.

Selfotel, also known as CGS-19755 and CPDD 0027, is a competitive NMDA antagonist. Selfotel directly competes with glutamate for binding to the receptor. Initial studies showed it to have anticonvulsant, anxiolytic, analgesic and neuroprotective effects, and it was originally researched for the treatment of stroke, but subsequent animal and human studies showed phencyclidine-like effects, as well as limited efficacy and evidence for possible neurotoxicity under some conditions, and so clinical development was ultimately discontinued.

Darodipine, also known as PY-108068, is a potent a calcium antagonist. Darodipine dose-depentently blocks I(Ca,L) in rat isolated cardiomyocytes. Darodipine exerts protective effects against free-radical-induced electrophysiological alterations independently of its calcium antagonistic properties; this effect is possibly due to trapping of specific radical species. .

Sunepitron, also known as CP-93,393 or CP-93,393-1 (HCl), is a combined 5-HT1A receptor agonist and α2-adrenergic receptor antagonist. It was previously under development by Pfizer for the treatment of depression and anxiety. It made it to phase III clinical trials before being discontinued.

Taribavirin, also known as Viramidine, is an antiviral drug candidate. It is a prodrug of ribavirin, active against a number of DNA and RNA viruses. Taribavirin has better liver-targeting than ribavirin, and has a shorter life in the body due to less penetration and storage in red blood cells. It is expected eventually to be the drug of choice for viral hepatitis syndromes in which ribavirin is active. These include hepatitis C and perhaps also hepatitis B and yellow fever.

Veralipride is a benzamide neuroleptic medicine indicated in the treatment of vasomotor symptoms associated with the menopause. It was first authorised for use in 1979. Veralipride was withdraw from the market in 2007.

Suxibuzone is a analgesic used for joint and muscular pain. It is a prodrug of the non-steroidal anti-inflammatory drug phenylbutazone, and is commonly used in horses.

Butibufen, also known as FF-106, is a cyclooxygenase inhibitor used to treat inflammatory and rheumatic disorders. utibufen was within the potency range of ibuprofen as an antipyretic and analgesic agent and greater than that of acetylsalicylic acid. The compound possesses the advantage of a low order to toxicity. Its ulcerogenicity was much lower than that of phenylbutazone. Therefore, butibufen appears to offer potential safety and efficacy in the treatment of rheumatic diseases. Butibufen was now withdraw from the market.

Alpidem is an anxiolytic drug from the imidazopyridine family, related to the more well known sleeping medication zolpidem. Unlike zolpidem however, alpidem does not produce sedative effects at normal doses, and is instead used specifically for the treatment of anxiety. It was approved for marketing in France in 1991 and was withdrawn fin 1994 and is not approved for marketing anywhere in the world. Alpidem has nanomolar binding affinity for both the central benzodiazepine receptor (CBR) and the peripheral benzodiazepine receptor (PBR) (translocator protein 18 kDa) (PBR (Ki ) 0.5-7 nM) and CBR (Ki ) 1-28 nM, respectively).

Medroxyprogesterone Acetate, also known as MPA, is a steroidal progestin, a synthetic variant of the steroid hormone progesterone. It is used as a contraceptive, in hormone replacement therapy and for the treatment of endometriosis as well as several other indications. MPA is a more potent derivative of its parent compound medroxyprogesterone (MP). While medroxyprogesterone is sometimes used as a synonym for medroxyprogesterone acetate, what is almost always being referred to is MPA and not MP It is on the World Health Organization's List of Essential Medicines, the most important medication needed in a basic health system.

Oxyphenbutazone, also known as G-29701, a non-steroidal anti-inflammatory drug (NSAID). It is a metabolite of phenylbutazone.